Juliette Siegfried, MPH

All articles by Juliette Siegfried, MPH

• Risk tolerance for gene therapy rises with DMD progression, survey finds

  A new study assessed the maximum risk of treatment-related death that people would accept to receive gene therapy for DMD.

• Patients and families value self-care, muscle and heart function in DMD treatment

  Researchers interviewed patients with DMD and caregivers to assess which key treatment characteristics matter most to them.

• DMD caregivers willing to tolerate risk in the hopes of prolonging ambulation

  A survey found that most DMD caregivers would choose a therapy with variable outcomes even if there was a risk of earlier loss of ambulation.

• Claims study finds costs rapidly increase as DMD progresses

  A German health care claims analysis finds that direct and indirect costs for DMD soar as patients progress through the disease stages.